Clinical Trial: A Phase II, Single Arm Study of BGJ398 in Patients With Advanced Cholangiocarcinoma

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Phase II Multicenter, Single Arm Study of Oral BGJ398 in Adult Patients With Advanced or Metastatic Cholangiocarcinoma With FGFR2 Gene Fusions or Other FGFR Genetic Alte

Brief Summary: This is a multi-center, open label, single arm phase II study evaluating BGJ398 anti-tumor activity in advanced or metastatic cholangiocarcinoma patients with FGFR genetic alterations.

Detailed Summary: Adult patients with histologically or cytologically confirmed advanced or metastatic cholangiocarcinoma with FGFR2 gene fusion/ translocation or other FGFR genetic alteration who have evidence of radiologic progression following a cisplatin-and gemcitabine-containing regimen for advanced disease or a gemcitabine-containing regimen for those who are considered intolerant to cisplatin will be enrolled. Approximately 55 adult patients over age 18, both male and female will be enrolled. Of these 55 patients, 40 patients will have cholangiocarcinoma with identified FGFR2 gene fusions and up to 15 patients may have other FGFR genetic alterations. All patients will receive oral BGJ398, once daily, on a three weeks on (21 days), one week off (7 days) schedule. One treatment cycle will consists of 28 days.
Sponsor: Novartis Pharmaceuticals

Current Primary Outcome: Overall response rate (ORR) [ Time Frame: up to 24 months ]

Overall response rate (ORR) is defined as the proportion of patients with a best overall response of Complete Response (CR) or Partial Response (PR), as per RECIST version 1.1.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Overall survival [ Time Frame: up to 24 months ]
    Overall Survival (OS) is defined as the time from the date of start of treatment to the date of death due to any cause.
  • Progression free survival [ Time Frame: up to 24 months ]
    Progression free survival (PFS) is defined as the date of the start of treatment to the date of the event defined as the first documented progression or death due to any cause.
  • Best overall response [ Time Frame: up to 24 months ]
    The best overall response is the best response recorded from the start of the treatment until disease progression.
  • Disease control rate [ Time Frame: up to 24 months ]
    Disease control rate (DCR) is the proportion of patients with a best overall response of CR or PR or SD.
  • Number of participants with adverse events (AEs) and serious adverse events (SAEs) as a measure of safety and tolerability [ Time Frame: up to 24 months ]
    To characterize the safety and tolerability of single agent BGJ398 by the type, frequency and severity of AEs & SAEs.
  • Selected trough and 2-hr Plasma concentration profile [ Time Frame: up to 12 months ]
    To determine selected trough and 2-hr plasma concentrations of BGJ398


Original Secondary Outcome: Same as current

Information By: Novartis

Dates:
Date Received: May 12, 2014
Date Started: July 14, 2014
Date Completion: June 29, 2020
Last Updated: March 17, 2017
Last Verified: March 2017